The health economics of orphan drugs: Testing a MCDA framework to enhance patient access

9th European Patients’ Rights Day, Brussels, May 2015

Carina Schey, Unit of Pharmacoepidemiology and Pharmacoeconomics

PhD Researcher

Supervisor: Prof. Dr. Maarten Postma

Health Economist at Global Market Access Solutions

• Increased demand on healthcare results in cost containment

initiatives

• Current health technology appraisals might disregard several key

disease attributes

• Orphan drugs are unlikely to achieve “accepted” cost/ QALY

• Results in poor access to orphan drugs in many countries

• New methods may facilitate reimbursement decision making

which will enhance the access by patients to life-sustaining drugs

Background

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• To develop a multi-criteria decision analysis model based on

the criteria and framework suggested by Hughes-Wilson et

al, 2012 (1)

• To assess the:

• Proposed criteria versus criteria highlighted in a

literature review

• Outcomes of the MCDA

Aims

1. Hughes-Wilson W, et al. Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? Orphanet Journal of Rare Diseases. 2012, 7: 74.

2

• Conducted a literature review

• Developed the MCDA framework

• Selection of study drugs

• Cost calculations

• Developed “scoring” system for different attributes

• Collected data for each criterion for each drug

Methods

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The proposed criteria

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• Disease severity• Level of impact on disease• Level of research undertaken• Unmet need• Manufacturing complexity• Follow up measures• Level of effectiveness uncertainty• Disease rarity• Uniqueness of the indication

Results: Individual criteria in relation to the overall drug score

PAH – Pulmonary arterial hypertension MPS II & VI – Mucopolysaccharidosis II & VIPNH – Paroxysmal nocturnal haemoglobinuria LGS – Lennox-Gastaut SyndromeMDS – Myelodysplastic syndromes

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Results: Average annual cost in relation to the total drug score

PAH – Pulmonary arterial hypertension MPS II & VI – Mucopolysaccharidosis II & VIPNH – Paroxysmal nocturnal haemoglobinuria LGS – Lennox-Gastaut SyndromeMDS – Myelodysplastic syndromes

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› Rational and transparent

› Includes a wide range of attributes

› Robust assessment

› Facilitates within-therapy and across-therapy comparisons

› Supports decision making

› Demonstrates true drug benefit and enhances patient access

where there is huge unmet need

Value of MCDAValue of MCDA

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› University of Groningen

Acknowledgements

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› University of Groningen

Acknowledgements

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